This year marks our 5th year of supporting research into therapies for the treatment of Parkinson’s disease in GBA mutation carriers. We are proud of our many accomplishments and collaborative relationships, which include making 39 grants to clinicians, scientists, academic laboratories, and biotechnology companies. We thank you for your ongoing interest.
Exciting New Grants in 2022
- A grant was made to Lysoway Therapeutics, a start-up biotech company with unique technological approaches to screen and develop potent modulators of lysosomal ion channels. Lysoway’s technology has the potential to re-balance lysosomal function and autophagy to treat neurodegenerative diseases, including Parkinson’s. Lysoway plans to use the grant to investigate the therapeutic potential of its channel modulators in preclinical models of GBA PD. The initial focus will be on target engagement and pharmacodynamic biomarkers.
- A grant was made to CuraSen Therapeutics, a company focused on the development of new treatments for neurodegenerative diseases. CuraSen’s drugs are designed to activate certain receptor populations in the brain to compensate for critical neuronal and glial functions that have otherwise been lost. The Phase 2 clinical trials will enroll patients with mild cognitive impairment or mild dementia due to either PD (with rapid eye movement sleep behavior disorder, PDRBD) or Alzheimer’s disease.
- In March, we provided funding to The New York Stem Cell Foundation (NYSCF) Research Institute to generate and study stem cell models of Parkinson’s disease from GBA-PD patients. NYSCF scientists will create induced pluripotent stem cell lines from a cohort of these patients. It will then use CRISPR gene editing to repair the GBA mutations, and convert both patient and gene-corrected stem cells into neurons. To discover entirely new features of GBA PD, NYSCF will also apply high-content imaging and artificial intelligence algorithms to pave the way for drug discovery.
- If genotyping has confirmed that you are a GBA carrier and you want to participate in this drug discovery effort, please contact Lisa Voltolina at [email protected] or [email protected]
- Our collaboration with The Michael J. Fox Foundation continued this year as we were proud to partner with MJFF to support a follow-up study from German biotech company MODAG. The goal of the technology developed by MODAG, is to stop the Parkinson’s protein alpha-synuclein (A-SYN) from clumping together. Scientists believe the aggregation of ASYN is toxic to brain cells. This funding for the expansion of a Phase 1B clinical trial in Parkinson’s disease will explore dosing strategies for future studies and the development of diagnostic tools including a PET tracer.
Updates on previous grants
- Aspen Neuroscience, Inc., which received a grant from The Silverstein Foundation in January 2019, announced a Series B funding round of $147.5 million in May. Aspen is a private biotechnology company developing autologous cell therapies, including the first iPSC-derived autologous neuron replacement treatment for Parkinson’s disease.
WISHING YOU ALL
A JOYFUL HOLIDAY SEASON AND A HEALTHY NEW YEAR